In late September, 2014, the FDA convened a public meeting entitled “Patient-Focused Drug Development for Hemophilia A, Hemophilia B, von Willebrand Disease and other Heritable Bleeding Disorders." Dr. Dana DiBenedetti, Head of Patient-Reported Outcomes, attended the meeting where she gleaned valuable insights that will support the work we do for manufacturers of drugs that treat these disorders.
The purpose of the meeting was to hear the “voice of the patient,” so that FDA can use it to provide clinical context for better assessing risks and benefits of new and existing products. According to the FDA, a patient is more willing to accept risks the more severe his or her condition, so a meeting like this provides the FDA with a better understanding of the real issues affecting patients and their caregivers.
Over 50 patients, caregivers, and advocates were present at the meeting (many more via WebEx) and many shared powerful stories about living and/or caring for loved ones with these disorders.
Dr. DiBenedetti said, “As a researcher focused on understanding patients’ perspectives, the meeting was invaluable. I heard detailed patient perspectives on symptoms and impacts of these disorders, unmet needs, drug safety and risk management issues, treatment costs, quality of life, treatment preferences, hurdles to accessing or adhering to treatment regimens, the effects of long-term and repetitive treatment, and misconceptions by health-care providers and how that leads to oversights and misdiagnoses."
Dr. DiBenedetti continued, “Clearly, these are complex disorders that require a patient-centric approach to treatment. A forum like this arms regulators, drug manufacturers, health care providers, and researchers like me with important data that can be used to inform the research and development of better treatment options. What I learned will directly benefit our clients who work to improve patients’ lives by developing novel treatments to address such conditions.”
Dr. DiBenedetti welcomes questions by manufacturers that are developing products to treat these disorders. She can be contacted here.